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Gene Therapy Trial Report

Summary

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis


NCTID NCT04273269 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication GM1 Gangliosidosis
Disease Ontology Term DOID:3322
Compound Name LYS-GM101
Sponsor LYSOGENE
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 5 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant GLB1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intracisternal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAVrh.10
Editor Type none
Dose 1 8E12 vg/kg (n=5)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2020-01-21
Completion Date 2023-05-22
Last Update 2023-06-09

Participation Criteria


Eligible Age <=3 Years
Standard Ages Child
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Documented GM1 gangliosidosis diagnosis based on genotyping confirming the β-gal gene mutations and/or documented deficiency of β-gal enzyme by laboratory testing * Children with early infantile GM1 gangliosidosis less than 12 months of age with ability to swallow * Children with late infantile GM1 gangliosidosis less than 3 years of age with ability to sit Exclusion Criteria: * Uncontrolled seizure disorder. Patients who are stable on anti-convulsive medications may be included * More than 40% brain atrophy as measured by MRI total brain volume at screening * Current participation in a clinical trial of another investigational medicinal product * Past participation in a gene therapy trial * History of hematopoietic stem cell transplantation * Any condition that would contraindicate treatment with immunosuppressant therapy * Presence of concomitant medical condition or anatomical abnormality precluding lumbar puncture or intracisternal injection * Presence of any permanent items (e.g., metal braces) precluding undergoing MRI * History of non-GM1 gangliosidosis medical condition that would confound scientific rigor or interpretation of results * Rare and unrelated serious comorbidities, e.g., Down syndrome, intraventricular hemorrhage in the new-born period, extreme low birth weight (\<1500 grams) or known bleeding disorders * Any vaccination 1 month prior to the planned immunosuppressant treatment * Serology consistent with HIV exposure or consistent with active hepatitis B or C infection * Grade 2 or higher lab abnormalities for Liver function tests (LFT), bilirubin, creatinine, hemoglobin, white blood cell (WBC) count, platelet count, prothrombin time (PT), and partial thromboplastin time (PTT), according to CTCAE v5.0
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 3
Locations United States,United Kingdom,France

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates 2 participants died due to progression of disease, little evidence of potential efficacy

Resources/Links